Who is conducting the clinical trial?
The University of Kansas Medical Center is conducting the study.
What is the purpose of the clinical trial?
Target FSHD is a Phase 1 study of a medicine called Clenbuterol. The purpose of the study is to:
How does the medicine work?
In FSHD, the DUX4 gene becomes active in muscles when it normally should be silent. Clenbuterol may help by turning down the harmful signals caused by DUX4 inside muscle cells. It works through beta‑2 receptors in muscle and is part of a group of medications called beta‑2 agonists.
Will some people be given a placebo instead of the medicine?
No, all patients will receive the medicine.
How will the medicine be given?
Clenbuterol will be taken orally twice daily.
How long is the clinical trial?
The trial will take 8-9 months with patients taking the medicine for 6 months.
What are the criteria to be included in the clinical trial?
You must:
The study team will assess you for:
What criteria would exclude me from the clinical trial?
You will not be able to take part if you:
Do I need to have a genetic test to be included? If so, will this be provided?
Yes, you must have a genetic test or you must have a first degree relative with a genetic report showing confirmation of FSHD1. If you don’t have one, the study team may be able to help you get one. However, the study will not cover the cost of your genetic test.
We recommend you work with your doctor to start the process of getting a genetic test before trying to join this clinical trial. Genetic testing can take many weeks to complete. Because of this, the study team may give priority to patients who are already tested, or who have already started the process. Learn more about genetic testing.
How will the study team determine whether I can take part in the study?
The first step is usually to provide medical records to the site for review. Next a pre-screening phone call to talk through the eligibility criteria will be completed bya coordinator at your study site. After this initial call, your coordinator may have additional pre-screening steps for you to complete. If you are a good fit, your coordinator will schedule an in-person screening visit.
Your screening visit will take place at a study site. At this visit, you will learn more about the study and provide your consent to proceed. Then, the study team will perform all the tests needed to determine if you can take part in the study. This could take several hours, or you may need multiple visits. After all your test results come back, the study team will tell you whether you can take part in study. This screening process can take up to 6 weeks.
What activities will I be asked to do?
How many study visits are there? Are they in-person or virtual?
You will be asked to attend 5 in-person visits and complete 1 safety follow-up call.
Will I have to stay overnight for any visits?
An overnight stay is not required for the study.
Will the medicine be offered to patients once the clinical trial is over?
No.
Will I have to pay for any medical expenses? Will my insurance be billed for any costs?
No, all costs are paid for by University of Kansas Medical Center.
Will I be reimbursed for travel costs?
Yes, reimbursement will be available for travel after approval of receipts.
Will I be paid a stipend for being in the clinical trial?
Yes, subjects will receive a $50 stipend for every on-site visit completed.
Who is paying for the clinical trial?
The study is funded by the National Institute of Arthritis and Musculoskeletal and Skin Disease through a research grant awarded at the University of Kansas Medical Center.
How many volunteers are needed?
The study needs 30 participants. However, many more volunteers are needed because not everyone who volunteers will be eligible and able to take part.
Where are the study sites that are conducting the clinical trial?
The study is conducted across multiple locations in the United States. To find specific sites, visit clinicaltrials.gov and click on “Contacts and Locations” in the sidebar to open up a list of locations. More locations may open as the study progresses.
How do I contact my study site to learn more and sign up?
To find specific sites, visit clinicaltrials.gov and click on “Contacts and Locations” in the sidebar to open up a list of locations.
If your study site is marked as “Recruiting,” there should be an email and/or phone number listed for contact. Sometimes there is also contact information listed for sites that are “Not Yet Recruiting.” You should email and call these contacts and let them know that you are interested in the Target FSHD study. If you are able and willing to travel to multiple different sites, you should contact each site individually.
What will happen after I contact my study site?
It can take several weeks or months to hear back, so be patient. Sites could still be in the process of getting ready for the study and may not be able to contact anyone just yet. Sometimes, sites are overloaded with more interested patients than they can enroll in the study. Make sure you provide your email and phone number to make it easier for the coordinators to contact you. You should also keep checking in on a monthly basis – this helps the study team know that you are committed to completing the clinical trial.
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